Paeds Vivas · haematology-oncology-and-transfusion
Thrombocytopenia and immune thrombocytopenia: Viva
Branching clinical structured oral on thrombocytopenia and immune thrombocytopenia in children, covering the diagnosis of isolated thrombocytopenia, the ASH 2019 observation-first principle, the first-line therapy with IVIG, corticosteroids, and anti-D, the second-line thrombopoietin-receptor agonists, and the rare splenectomy, appraising the ICIS registries, the Blanchette anti-D trial, and the PETIT eltrombopag trial.
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Target exams
This is the classic presentation of newly diagnosed immune thrombocytopenia in a well preschool child. The structured oral runs from the diagnostic criteria through the observation decision, the first-line therapy if the bleeding worsened, the second-line strategy, and the trial evidence. [1][4]
Opening: make the diagnosis
Examiner. The platelet count is 12 times ten to the nine per litre. What is the diagnosis, and how do you confirm it? [4]
Candidate. This is immune thrombocytopenia, ITP, defined by the Rodeghiero standard as an isolated thrombocytopenia with a platelet count under 100 times ten to the nine per litre, in an otherwise well child, with a normal blood film showing large platelets and no blasts, and no hepatosplenomegaly or lymphadenopathy. The diagnosis is clinical and is made by exclusion. The normal haemoglobin and white cell count, the large platelets and the absence of blasts on the film, the well child, and the absence of organomegaly all fit. A bone marrow aspirate is not indicated, because the ASH 2019 guideline recommends against the routine marrow aspirate in the typical presentation. [1][4]
Examiner (probe). What would make you doubt the diagnosis? [3]
Candidate. The features that exclude ITP are the other cytopenias, the anaemia or the neutropenia, the blast cells on the film, the hepatosplenomegaly or the significant lymphadenopathy, and the fever or the bone pain. Any of these points to the acute leukaemia or another infiltrative disorder, and I would order the bone marrow aspirate. The small platelets would point to the Wiskott-Aldrich syndrome, and the schistocytes would point to the haemolytic uraemic syndrome. [3]
References5ShowHide
- [1]Neunert C, Terrell DR, Arnold DM American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv, 2019.PMID 31794604
- [3]Cooper N, Ghanima W Immune Thrombocytopenia. N Engl J Med, 2019.PMID 31483965
- [4]Rodeghiero F, Stasi R, Gernsheimer T Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood, 2009.PMID 19005182
- [8]Blanchette V, Imbach P, Andrew M Randomised trial of intravenous immunoglobulin G, intravenous anti-D, and oral prednisone in childhood acute immune thrombocytopenic purpura. Lancet, 1994.PMID 7915773
- [10]Bussel JB, de Miguel PG, Despotovic JM Eltrombopag for the treatment of children with persistent and chronic immune thrombocytopenia (PETIT): a randomised, multicentre, placebo-controlled study. Lancet Haematol, 2015.PMID 26688484